A new strategy to enhance gene therapy for sickle cell disease
Researchers described a promising new approach for using gene therapy to treat sickle cell disease in the journal Human Gene ...
FierceBiotech2d
CSL Behring shuts down California cell and gene therapy R&D site amid group pivot
In another sign of biopharma's waning interest in the traditional ex vivo gene therapy approach, CSL Behring is shutti | CSL ...
WebMD1d
First Brain-Injected Gene Therapy Approved by FDA
The FDA has granted fast-track approval for a groundbreaking gene therapy indicated for a rare genetic disorder called ...
FierceBiotech1d
Neurogene stops Batten gene therapy after FDA denies request
Talks with the FDA haven’t gone according to plan. Seeking a streamlined pathway, Neurogene submitted a Regenerative Medicine ...
BioSpace23h
Neurogene Drops Batten Gene Therapy Program
Failing to receive the RMAT designation from the FDA for its early-stage Batten disease gene therapy, Neurogene tells ...
Rocket Pharmaceuticals' Gene Therapy Progress Boosts Confidence in Future Developments: Analyst
On Monday, Rocket Pharmaceuticals, Inc. (NASDAQ:RCKT) presented long-term safety and efficacy results from the Phase 1 study ...
MedPage Today on MSN1d
Gene Therapy Promising for Rare Genetic Cardiomyopathy
CHICAGO -- Gene therapy for Danon disease, a rare inherited cause of hypertrophic cardiomyopathy, appeared to improve or ...
STAT2d
Regenxbio says gene therapy strengthened boys with Duchenne muscular dystrophy
Based on a small trial with positive muscle performance data, Regenxbio is expected to pursue approval of its gene therapy ...
Pharmaceutical Technology2d
Intellia’s gene editing therapy shows early potential in rare heart condition
Intellia presented data from its Phase I study of NTLA-2001 at the 2024 American Heart Association scientific meeting in ...
Medpage Today on MSN6d
First Brain-Delivered Gene Therapy Approved for AADC Deficiency
The treatment is the first approved gene therapy for AADC deficiency, the FDA said. People with this rare disorder have ...
Science Daily5d
New discovery enables gene therapy for muscular dystrophies, other disorders
StitchR, a new gene therapy technique, delivers large genes in two parts to treat muscular dystrophies by restoring critical proteins in animal models.
Taiwan News2h
Voyager Selects Tau Silencing Gene Therapy Development Candidate for Alzheimer’s Disease
- VY1706 is the fifth neuro gene therapy development candidate nominated leveraging Voyager’s IV-delivered, CNS-penetrant TRACER capsids - ...